A new approach to cholesterol lowering that targets a regulator of harmful Low-density lipoprotein (LDL) cholesterol could offer new treatment options for patients with high cholesterol, researchers said in a study published in The Lancet.

The drug, called ALN-PCS, blocks production of the cholesterol regulator PCSK9, a protein that destroys receptors that normally clear harmful cholesterol from the blood.

In a trial involving 32 healthy volunteers with mildly to moderately raised LDL, or 'bad' cholesterol, people were randomly assigned to receive either an injection of ALN-PCS or saline as a placebo.
A single dose of ALN-PCS was shown to cut levels of 'bad' cholesterol by up to 57 percent, researchers said.

"People with extremely high cholesterol are at increased risk of a heart attack and this approach could offer new hope for those who are resistant to statins," British Heart Foundation Medical Director, Professor Peter Weissberg, said.

"These initial results add to growing evidence that blocking the action of a certain protein can dramatically lower 'bad' LDL cholesterol. More research is now needed to confirm this approach is both safe and effective at preventing heart attacks in the long term before it becomes widely available," said Weissberg.


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